First person treated with cell-rejuvenating gene therapy for glaucoma

Life Biosciences Doses First Patient in Anti-Aging Gene Therapy Trial for Glaucoma

Life Biosciences on Monday announced that the first participant has been dosed in its Phase 1 clinical trial of ER-100, a gene therapy designed to reverse cellular aging in patients with vision loss — marking the first time partial epigenetic reprogramming has been tested in a human.globenewswire

The Boston-based biotechnology company, co-founded by Harvard Medical School biologist David Sinclair, is testing whether delivering three transcription factors — OCT4, SOX2, and KLF4, collectively known as OSK — can rejuvenate deteriorating retinal ganglion cells in patients with open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION).nature

How the Therapy Works

ER-100 uses viral vectors to deliver genetic material directly into retinal cells. A second viral vector, activated by the antibiotic doxycycline, acts as a safety switch: when patients take a doxycycline pill, the therapy turns on; when they stop, it turns off. Participants will take doxycycline for eight weeks alongside a steroid to reduce inflammation.nad

The approach aims to reset epigenetic patterns — the chemical tags on DNA that accumulate with age — to a more youthful state, restoring function in damaged neurons that are not normally capable of regeneration. Life Biosciences reported in 2024 that the therapy restored visual function in a primate model of NAION.linkedin

Safety Questions Linger

The trial will primarily assess safety and tolerability, a lingering concern in the reprogramming field. The technique uses three of the four Yamanaka factors that, in laboratory settings, can push adult cells all the way back to a stem-cell-like state. The goal is to nudge cells back only partway — enough to rejuvenate without causing them to lose their specialized identity. Some scientists have cautioned that reprogramming gone awry could trigger uncontrolled cellular growth, a risk observed in earlier mouse studies.nature

A Landmark for the Longevity Field

The FDA cleared Life Biosciences’ Investigational New Drug application on January 15, 2026, making ER-100 the first cellular rejuvenation therapy using epigenetic reprogramming to enter human trials. The company raised $80 million in Series D financing in April 2026 to fund the trial’s completion and advance its broader platform into other tissue types, bringing total funding to nearly $240 million.lifebiosciences

The trial will enroll up to six glaucoma patients at two dose levels before establishing a dose to test in up to six NAION patients, and is being conducted at Harvard-affiliated Massachusetts Eye and Ear in Boston.nad

“The goal is to restore vision to some level and prevent further vision loss,” Life Biosciences CEO Jerry McLaughlin said when the FDA clearance was announced earlier this year.nad